Phase III Study of L-Cysteine in Patients With Erythropoietic Protoporphyria

This study is ongoing, but not recruiting participants.

First Received: February 24, 2000 Last Updated: June 23, 2005 History of Changes

Sponsor:	FDA Office of Orphan Products Development
Collaborator:	Brigham and Women's Hospital
Information provided by:	FDA Office of Orphan Products Development
ClinicalTrials.gov Identifier:	NCT00004940

Purpose

OBJECTIVES:

I. Determine the long-term efficacy and safety of L-cysteine in the prevention photosensitivity in patients with erythropoietic protoporphyria.

Condition	Intervention	Phase
Erythropoietic Protoporphyria	Drug: cysteine hydrochloride	Phase III

Study Type:	Interventional
Study Design:	Treatment

Resource links provided by NLM:

Genetics Home Reference related topics: porphyria

Drug Information available for: Cysteine Cysteine hydrochloride

U.S. FDA Resources

Further study details as provided by FDA Office of Orphan Products Development:

Estimated Enrollment:	50
Study Start Date:	May 1996
Estimated Study Completion Date:	May 1996

Detailed Description:

PROTOCOL OUTLINE: This is a phase III study, lasting 3 years; 1996-1999. Patients are administered L-cysteine orally twice daily, 2 capsules with breakfast and 2 with lunch.

Patients fill in questionnaires and diary sheets about their reaction to sunlight exposure, and have blood tested 3 times a year.

Eligibility

Ages Eligible for Study:	18 Years to 65 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

PROTOCOL ENTRY CRITERIA:

--Disease Characteristics--

Erythropoietic protoporphyria RBC protoporphyrin greater than 50 micrograms/100 dL

--Prior/Concurrent Therapy--

At least 3 months since prior betacarotene or L-cysteine

No concurrent betacarotene

--Patient Characteristics--

Fertile female patients must use effective contraception for duration of trial and for 3 weeks thereafter
Not pregnant or nursing

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00004940

Sponsors and Collaborators

FDA Office of Orphan Products Development

Brigham and Women's Hospital

Investigators

Study Chair:

Micheline Mary Mathews-Roth

Brigham and Women's Hospital

More Information

No publications provided

Study ID Numbers:	199/13376, BWH-FDR000996-DR
Study First Received:	February 24, 2000
Last Updated:	June 23, 2005
ClinicalTrials.gov Identifier:	NCT00004940 History of Changes
Health Authority:	United States: Federal Government

Keywords provided by FDA Office of Orphan Products Development:

erythropoietic protoporphyria
inborn errors of metabolism
porphyria
rare disease

Additional relevant MeSH terms:

Metabolism, Inborn Errors
Liver Diseases
Metabolic Diseases
Digestive System Diseases
Skin Diseases
Genetic Diseases, Inborn

Porphyrias
Skin Diseases, Metabolic
Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Skin Diseases, Genetic

ClinicalTrials.gov processed this record on November 30, 2009